An experimental new treatment improves survival and quality of life among patients with a severe form of an autoimmune disease called scleroderma, researchers report in the New England Journal of Medicine. The disease, called scleroderma, causes the skin and connective tissues to harden and, in its most severe form, can harden internal organs and lead to death. “It’s really a devastating and mortal disease,” study author Dr. Keith Sullivan of Duke tells me.
Sullivan and his colleagues tested a treatment in which patients first undergo chemotherapy and radiation to destroy their bone marrow, then are given a transplant of their own blood-forming stem cells to replace it. “It’s starting from scratch,” Sullivan says. Patients on the experimental regimen were less likely to die due to the progression of their disease than patients who were given an immune-suppressing drug sometimes used to treat the disease. The treatment did, however, carry short-term risks including infection and low blood cell counts, as well as an increased risk of a secondary cancer due to the chemo and radiation.